TORONTO, March 26, 2013 /CNW/ - As part of the Common Drug Review
process, the Canadian Drug Expert Committee (CDEC) has recommended that
the drug KALYDECO (ivacaftor) be listed on the formulary listing of
publicly funded drug plans for the treatment of cystic fibrosis in
patients age six years and older who have the G551D mutation in the
Cystic Fibrosis Transmembrane conductance Regulator.

"On behalf of cystic fibrosis patients and their families, we welcome
the recommendation of the Canadian Drug Expert Committee," said Maureen
Adamson, President and CEO of Cystic Fibrosis Canada. "We are grateful
for the opportunity to make a patient input submission as part of the
Common Drug Review process and are pleased that the voices of our
patients and their families were listened to. I look forward to even
better news when this drug will be accessible to the patients who will
benefit from it."

KALYDECO is the first therapy that targets the underlying cause of
cystic fibrosis. This drug helps to improve the function of the
defective protein, leading to better lung function, weight gain and
lower sweat chloride levels. For a CF patient with the specific G551D
mutation, access to KALYDECO could lead to a healthier, longer life.
The G551D mutation is present in roughly 100 Canadians with cystic
fibrosis.

"While this announcement is positive news, our efforts will now focus on
asking provincial governments across the country to fund this drug as
soon as possible," said Ken Chan, Vice President of Advocacy, Research
and Healthcare at Cystic Fibrosis Canada. "We will be calling on
provincial health ministers to join their counterparts in the United
States, United Kingdom and Ireland in providing access to this
breakthrough drug."

In October 2012, Cystic Fibrosis Canada made a submission to the Common
Drug Review (CDR) process on the drug KALYDECO. Testimonials submitted
for affected patients who are currently being treated with KALYDECO
were compelling. They report transitions from debilitating illness and
exhaustion to having more energy, being able to finally exercise, being
able to spend more time with their families, and an overall enhanced
quality of life. Many patients strongly believe that using KALYDECO
will allow them to avoid lung transplantation for end-stage lung
disease.

Background: Access to KALYDECO in Canada

Of the 4,000 Canadians living with this devastating disease, about 100
CF patients in Canada have the specific G551D mutation of the CFTR gene
that can be treated by KALYDECO. (Source: Canadian CF Registry). When
taken twice a day with fat-containing food, KALYDECO helps the protein
made by the CFTR gene function better, improving lung function and
weight gain. (Source: FDA)

The Common Drug Review (CDR) was established by the federal, provincial,
and territorial government to provide publicly-funded drug plans
(except Quebec) with high-quality drug reviews that will inform their
decision making.

KALYDECO has been available to patients in the United States through
private insurance or state Medicaid plans since 2012. In 2013, England,
Scotland, Ireland and Northern Ireland started funding KALYDECO for
use by their patients.

Cystic fibrosis is the most common fatal genetic disease affecting
Canadian children and young adults. It is a multi-system disease that
affects mainly the lungs and the digestive system. In the lungs, where
the effects are most devastating, a build-up of thick mucus causes
severe respiratory problems. Mucus and protein also build up in the
digestive tract, making it difficult to digest and absorb nutrients
from food. As improved therapies have helped to address the
malnutrition issues, ultimately most deaths related to cystic fibrosis
are due to lung disease. Currently, there is no cure.

Cystic Fibrosis Canada

Cystic Fibrosis Canada is one of the world's top three charitable
organizations committed to finding a cure for cystic fibrosis and is an
internationally-recognized leader in funding CF research, innovation,
and clinical care. We invest more funding in life-saving CF research
and care than any other non-governmental agency in Canada. Since 1960,
Cystic Fibrosis Canada has invested more than $140 million in leading
research and care, resulting in one of the world's highest survival
rates for Canadians living with cystic fibrosis. For more information,
visit www.cysticfibrosis.ca.